Accelerating Drug Development for Neuroblastoma: Consensus Statement From the Third Neuroblastoma Drug Development Strategy Forum

作者：Steven G. DuBois, Lucas Moreno, John Anderson, Shahab Asgharzadeh, Rochelle Bagatell, Maja Beck Popovic, J. S. Van Belle, Pablo Berlanga, Nick Bird, Louis Chesler, Adam David Durbin, Angelika Eggert, Martin Eilers, Sara Michele Federico, Matthias Fischer, Susanne A. Gatz, Rani E. George, Sally L. George, Kelly C. Goldsmith, Juliet C. Gray, Andras Attila Heczey, Meredith S. Irwin, Leona Knox, Holger N. Lode, Donna Ludwinski, Margaret E. Macy, Robbie G. Majzner, John M. Maris, Shakeel I. Modak, Jan J. Molenaar, Daniel A. Morgenstern, Yaël P. Mossé, Cormac Owens, C. Patrick Reynolds, Claudia Rössig, Gudrun Schleiermacher, Liz Scott, Paul M. Sondel, Frank Speleman, Max M. van Noesel, Frank Westermann, Judith Wienke, Adam J. Wolpaw, Julie R. Park, Andrew D.J. Pearson · 发表于：Pediatric Blood & Cancer · 年份：2025 · DOI：10.1002/pbc.31831 · 被引用次数：13 · 研究领域：Neuroblastoma Research and Treatments、Cancer therapeutics and mechanisms、Cancer, Hypoxia, and Metabolism

High-risk neuroblastoma is a poor prognosis cancer of the sympathetic nervous system that accounts for a disproportionate number of childhood cancer deaths. Many viable biological targets have been identified, and the number of potential combinations is even larger. Several products have attained marketing authorization for treatment of patients with neuroblastoma. Patient outcomes remain poor, with approximately 50% of children with newly diagnosed high-risk neuroblastoma cured of their disease. International, multistakeholder Neuroblastoma Drug Development Strategy (NDDS) meetings were established more than a decade ago. This third NDDS meeting included academia, industry, regulatory, and patient advocacy representatives to prioritize agents and to address key challenges in drug development in this disease. Given the central role that anti-GD2 therapy plays, novel GD2-directed combinations were a key focus, including epigenetic enzymes such as EZH2 and immunologic targets such as IL15 and TIGIT as potential combination partners. GD2-directed chimeric antigen receptor (CAR)-T cells were a top priority, along with emerging CAR-T targets such as B7-H3 and GPC2. Recognizing that combination therapies are likely to be most impactful for patients and for advancing therapies to frontline, another key focus was on high priority combinations of targeted therapies, including Aurora A kinase plus BCL2 or ATR inhibitors. Additional targets and agents were prioritized or deprioritized b...